Made in Chi­na: TaiMed wins FDA OK for a 'break­through' HIV ther­a­py for drug-re­sis­tant pa­tients

The FDA has hand­ed TaiMed and its part­ners at Ther­at­e­ch­nolo­gies an ap­proval for the first new HIV drug to come along in a decade with a nov­el mech­a­nism of ac­tion.

The drug, to be mar­ket­ed as Trog­a­r­zo (ibal­izum­ab-uiyk), works by bind­ing to the sec­ond ex­tra­cel­lu­lar do­main of the CD4+ T cell re­cep­tor, away from oth­er bind­ing sites used by drugs al­ready on the mar­ket. That ap­proach al­lows pa­tients who are be­com­ing re­sis­tant to their ther­a­pies to add this to the mix be­fore be­ing ex­posed to a lethal amount of virus.

Tai­wan-based TaiMed of­fered enough da­ta from a mid-stage pro­gram to war­rant both a break­through ther­a­py des­ig­na­tion as well as a pri­or­i­ty re­view for this drug, slic­ing the time need­ed for an FDA in­spec­tion.

For a low pro­file ther­a­py, this drug is a re­mark­able trend­set­ter in many ways. Shang­hai-based WuXi Bi­o­log­ics — part of Ge Li’s ex­pan­sive bio­phar­ma em­pire — will man­u­fac­ture the drug, and the FDA com­plet­ed its first-ever in­spec­tion and ap­proval of a Chi­nese man­u­fac­tur­ing fa­cil­i­ty to lay the foun­da­tion for the com­mer­cial roll­out.

“While most pa­tients liv­ing with HIV can be suc­cess­ful­ly treat­ed us­ing a com­bi­na­tion of two or more an­ti­retro­vi­ral drugs, a small per­cent­age of pa­tients who have tak­en many HIV drugs in the past have mul­tidrug re­sis­tant HIV, lim­it­ing their treat­ment op­tions and putting them at a high risk of HIV-re­lat­ed com­pli­ca­tions and pro­gres­sion to death,” said Jeff Mur­ray, deputy di­rec­tor of the Di­vi­sion of An­tivi­ral Prod­ucts in the FDA’s CDER. “Trog­a­r­zo is the first drug in a new class of an­ti­retro­vi­ral med­ica­tions that can pro­vide sig­nif­i­cant ben­e­fit to pa­tients who have run out of HIV treat­ment op­tions. New treat­ment op­tions may be able to im­prove their out­comes.”

TaiMed out-li­censed the drug to Mon­tre­al-based Ther­at­e­ch­nolo­gies a cou­ple of years ago in a deal that starts very small. The biotech bagged just a mil­lion dol­lars, ini­tial­ly, with an­oth­er mil­lion due on launch. There’s a few mil­lion more in shares for var­i­ous con­di­tions, and $10 mil­lion once an­nu­al sales of $200 mil­lion are reached in the US. That goes up to a $100 mil­lion if they can break the $1 bil­lion mark.

Ther­at­e­ch­nolo­gies al­so agreed to a de­vel­op­ment mile­stone for Phase III of $50 mil­lion, paid quar­ter­ly.

In­vestors liked the sound of all of it. Ther­at­e­ch­nolo­gies’ shares $THERF soared 30% on the news.

De­vel­op­ment of the Next Gen­er­a­tion NKG2D CAR T-cell Man­u­fac­tur­ing Process

Celyad’s view on developing and delivering a CAR T-cell therapy with multi-tumor specificity combined with cell manufacturing success
Transitioning potential therapeutic assets from academia into the commercial environment is an exercise that is largely underappreciated by stakeholders, except for drug developers themselves. The promise of preclinical or early clinical results drives enthusiasm, but the pragmatic delivery of a therapy outside of small, local testing is most often a major challenge for drug developers especially, including among other things, the manufacturing challenges that surround the production of just-in-time and personalized autologous cell therapy products.

Roger Perlmutter, Merck

#ASH19: Here’s why Mer­ck is pay­ing $2.7B to­day to grab Ar­Qule and its next-gen BTK drug, lin­ing up Eli Lil­ly ri­val­ry

Just a few months after making a splash at the European Hematology Association scientific confab with an early snapshot of positive data for their BTK inhibitor ARQ 531, ArQule has won a $2.7 billion buyout deal from Merck.

Merck is scooping up a next-gen BTK drug — which is making a splash at ASH today — from ArQule in an M&A pact set at $20 a share $ARQL. That’s more than twice Friday’s $9.66 close. And Merck R&D chief Roger Perlmutter heralded a deal that nets “multiple clinical-stage oral kinase inhibitors.”

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Game on: Re­gen­eron's BC­MA bis­pe­cif­ic makes clin­i­cal da­ta de­but, kick­ing off mul­ti­ple myelo­ma matchup with Bris­tol-My­ers

As J&J attempts to jostle past Bristol-Myers Squibb and bluebird for a landmark approval of its anti-BCMA CAR-T — and while GlaxoSmithKline maps a quick path to the FDA riding on its own BCMA-targeting antibody-drug conjugates — the bispecifics are arriving on the scene to stake a claim for a market that could cross $10 billion per year.

The main rivalry in multiple myeloma is shaping up to be one between Regeneron and Bristol-Myers, which picked up a bispecific antibody to BCMA through its recently closed $74 billion takeover of Celgene. Both presented promising first-in-human data at the ASH 2019 meeting.

FDA lifts hold on Abeon­a's but­ter­fly dis­ease ther­a­py, paving way for piv­otal study

It’s been a difficult few years for gene and cell therapy startup Abeona Therapeutics. Its newly crowned chief Carsten Thiel was forced out last year following accusations of unspecified “personal misconduct,” and this September, the FDA imposed a clinical hold on its therapy for a form of “butterfly” disease. But things are beginning to perk up. On Monday, the company said the regulator had lifted its hold and the experimental therapy is now set to be evaluated in a late-stage study.

Paul Hudson. Sanofi

New Sanofi CEO Hud­son adds next-gen can­cer drug tech to the R&D quest, buy­ing Syn­thorx for $2.5B

When Paul Hudson lays out his R&D vision for Sanofi tomorrow, he will have a new slate of interleukin therapies and a synthetic biology platform to boast about.

The French pharma giant announced early Monday that it is snagging San Diego biotech Synthorx in a $2.5 billion deal. That marks an affordable bolt-on for Sanofi but a considerable return for Synthorx backers, including Avalon, RA Capital and OrbiMed: At $68 per share, the price represents a 172% premium to Friday’s closing.

Synthorx’s take on alternative IL-2 drugs for both cancer and autoimmune disorders — enabled by a synthetic DNA base pair pioneered by Scripps professor Floyd Romesberg — “fits perfectly” with the kind of innovation that he wants at Sanofi, Hudson said.

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Roche faces an­oth­er de­lay in strug­gle to nav­i­gate Spark deal past reg­u­la­tors — but this one is very short

Roche today issued the latest in a long string of delays of its $4.3 billion buyout of Philadelphia-based Spark Therapeutics. The delay comes as little surprise — it is their 10th in as many months — as their most recent delay was scheduled to expire before a key regulatory deadline.

But it is notable for its length: 6 days.

Previous extensions had moved the goalposts by about 3 weeks to a month, with the latest on November 22 expiring tomorrow. The new delay sets a deadline for next Monday, December 16, the same day by which the UK Competition and Markets Authority has to give its initial ruling on the deal. And they already reportedly have lined up an OK from the FTC staff – although that’s only one level of a multi-step process.

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KalVis­ta's di­a­bet­ic mac­u­lar ede­ma da­ta falls short — will Mer­ck walk away?

Merck’s 2017 bet on KalVista Pharmaceuticals may have soured, after the UK/US-based biotech’s lead drug failed a mid-stage study in patients with diabetic macular edema (DME).

Two doses of the intravitreal injection, KVD001, were tested against a placebo in a 129-patient trial. Patients who continued to experience significant inflammation and diminished visual acuity, despite anti-VEGF therapy, were recruited to the trial. Typically patients with DME — the most frequent cause of vision loss related to diabetes — are treated with anti-VEGF therapies such as Regeneron’s flagship Eylea or Roche’s Avastin and Lucentis.

UP­DAT­ED: Ob­sE­va makes case for best-in-class hor­mone sup­pres­sive ther­a­py in pos­i­tive uter­ine fi­broid study

About a month after the Swiss biotech disclosed a failed late-stage study in its IVF program, ObsEva on Monday unveiled positive pivotal data on its experimental treatment for heavy menstrual bleeding triggered by uterine fibroids.

ObsEva in-licensed the drug, linzagolix, from Japan’s Kissei Pharmaceutical in 2015. Two doses of the drug (100 mg and 200 mg) were tested against a placebo in the 535-patient Phase III study, dubbed PRIMROSE 2, in patients who were both on and off hormonal add-back therapy (ABT).

Ear­ly-stage can­cer biotech nails $85M C round; Flem­ming Orn­skov's Gal­der­ma scores 'break­through' sta­tus

→ Zentalis Pharmaceuticals just nabbed an $85 million round from a syndicate that includes Matrix Capital, Viking Global Investors, Redmile Group, Farallon Capital, Perceptive Advisors, Surveyor Capital and Eventide Asset Management. Their lead drug is ZN-c5, which is currently in Phase I/II trials. The biotech describes that drug as a “potential best-in-class oral Selective Estrogen Receptor Degrader for estrogen receptor-positive, HER2-negative (ER+/ HER2-) breast cancer.”